Cellatica

Researchers are evaluating the efficacy and safety of empasiprubart in adults with Chronic Inflammatory Demyelinating Polyneuropathy CIDP. This Phase 3, randomized, double-blinded, placebo-controlled study compares empasiprubart to placebo to better understand its impact on CIDP symptoms and disease progression. The study has two parts Part A lasts 24 weeks 6 months, where participants receive either empasiprubart or placebo via intravenous infusion. After Part A, all participants enter Part B for 96 weeks 24 months during which everyone receives empasiprubart. Participants who received empasiprubart in Part A will receive a placebo dose once during Part B to maintain the study blind. Participants will have regular assessments including measurements of disability using the adjusted inflammatory neuropathy cause and treatment aINCAT score, grip strength, and other neurological and quality of life scales. Safety is monitored throughout the study. The total participation period spans up to 120 weeks, with evaluations at multiple time points to track changes from baseline and any adverse events.

Researchers are evaluating the effectiveness of claseprubart DNTH103 compared to a placebo in adults with chronic inflammatory demyelinating polyneuropathy CIDP. This Phase 3 study aims to assess treatment outcomes in participants with typical CIDP or certain CIDP variants, focusing on improving disease activity and disability measures. The study consists of several periods Part A includes an open-label phase lasting up to 13 weeks where participants receive an intravenous loading dose of claseprubart followed by subcutaneous injections every two weeks. Part B is a randomized, placebo-controlled, double-blind treatment phase lasting up to 52 weeks for those who respond to treatment in Part A, with participants receiving either claseprubart or placebo subcutaneously every two weeks. Eligible participants may then join an optional open-label extension lasting up to 104 weeks, continuing claseprubart treatment subcutaneously every two weeks, followed by a safety follow-up period of 40 weeks. Participants will undergo regular assessments throughout the study, including evaluations of disease relapse using the Adjusted Inflammatory Neuropathy Cause and Treatment INCAT score, disability scales, grip strength measurements, quality of life, fatigue severity, and antibody levels. Safety monitoring involves tracking adverse events and drug serum concentrations. The total study duration can extend up to approximately 209 weeks, including all treatment and follow-up phases, with careful monitoring of participants neurological stability and treatment responses.

Researchers are evaluating the effectiveness and safety of masitinib combined with riluzole compared to a placebo combined with riluzole for treating Amyotrophic Lateral Sclerosis ALS. This phase 3 study focuses on patients diagnosed with probable or definite ALS, aiming to understand how masitinib, a drug that targets cells involved in neuroinflammation, might slow disease progression and affect the nervous systems environment. Participants receive oral masitinib at 3.0 mgkgday twice daily, with dose increases to 4.5 mgkgday after 4 weeks, and for some, an additional increase to 6.0 mgkgday after another 4 weeks. Each dose increase includes safety monitoring. Masitinib is given alongside riluzole, a standard ALS treatment at 50 mg twice daily. The control group receives a matching placebo with riluzole. The study is randomized, double-blind, and includes two different masitinib dose escalation schedules. During the 48-week study period, participants undergo regular assessments including functional rating scales ALSFRS-R, quality of life questionnaires ALSAQ-40, survival and progression monitoring, lung function tests FVC, and muscle strength measurements HHD. Researchers measure changes in combined function and survival scores. Safety is closely monitored throughout, with follow-up extending up to 36 months for disease progression or death. Total participation includes baseline screening, treatment, and long-term observation.

This research aims to collect detailed information about Pompe disease, a rare genetic disorder also known as Glycogen Storage Disease Type II. The study is a global, long-term observational program designed to better understand the diseases progression, variability, and identification in patients who are either treated or untreated. It also supports regulatory requirements, product development, reimbursement, and other research purposes. Participants in the Pompe Registry are tracked over many years, up to 30 years, to observe the natural history of the disease and evaluate long-term outcomes, including the effects of treatments like alglucosidase alfa. This observational study does not involve experimental treatments but gathers data from patients worldwide to improve care strategies and recommendations. During the study, participants health information is collected retrospectively and prospectively, including clinical outcomes and disease manifestations. Researchers analyze these data to understand patient variability, disease progression, and treatment effectiveness. The registry helps develop guidance for monitoring patients and provides valuable insights to optimize Pompe disease care over an extended period.

Researchers are evaluating SRP-1003 in adults aged 18 to 65 with type 1 myotonic dystrophy DM1 in this phase 12a study. The trial aims to assess the safety, tolerability, how the drug moves through and affects the body pharmacokinetics and pharmacodynamics, comparing different doses of SRP-1003 to a placebo. Participants must have genetically confirmed DM1 with symptoms starting after age 12 and show clinical signs including myotonia. Participants will be randomly assigned to receive either SRP-1003 or a placebo through intravenous IV infusion or subcutaneous SC injection. The study has two parts Part 1 involves single doses, and Part 2 involves multiple doses. The treatment will be given under close monitoring to evaluate how the drug is processed and its effects. During the study, participants will have assessments including monitoring for adverse events up to 90 days for single-dose and 180 days for multiple-dose phases. Researchers will measure drug levels in the blood, changes in motor function tests such as hand opening time, timed walking tests, muscle strength, and quality of life scales specific to DM1. Safety and tolerability will be carefully observed throughout the study period that lasts several months.